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WO1997011169A3 - Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed - Google Patents

Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed Download PDF

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Publication number
WO1997011169A3
WO1997011169A3 PCT/GB1996/002357 GB9602357W WO9711169A3 WO 1997011169 A3 WO1997011169 A3 WO 1997011169A3 GB 9602357 W GB9602357 W GB 9602357W WO 9711169 A3 WO9711169 A3 WO 9711169A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene
strategy
coding regions
expression
bind
Prior art date
Application number
PCT/GB1996/002357
Other languages
French (fr)
Other versions
WO1997011169A2 (en
Inventor
Gwenyth Jane Farrar
Peter Humphries
Paul Francis Kenna
Original Assignee
Trinity College Dublin
Gwenyth Jane Farrar
Peter Humphries
Paul Francis Kenna
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Trinity College Dublin, Gwenyth Jane Farrar, Peter Humphries, Paul Francis Kenna filed Critical Trinity College Dublin
Priority to DE69637831T priority Critical patent/DE69637831D1/en
Priority to US09/043,506 priority patent/US6713457B2/en
Priority to EP96931887A priority patent/EP0851918B1/en
Priority to CA2232738A priority patent/CA2232738C/en
Priority to AU70896/96A priority patent/AU7089696A/en
Priority to NZ318818A priority patent/NZ318818A/en
Publication of WO1997011169A2 publication Critical patent/WO1997011169A2/en
Publication of WO1997011169A3 publication Critical patent/WO1997011169A3/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/78Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121Hammerhead
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/15Nucleic acids forming more than 2 strands, e.g. TFOs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)

Abstract

The invention provides strategy for suppressing expression of an endogenous gene, wherein said strategy comprises providing suppression effectors able to bind to the non-coding regions of a gene to be suppressed, to prevent the functional expression thereof. The suppression effectors may be antisense nucleic acids, and the non-coding regions can include the transcribed but non-translated regions of a gene. The strategy can also introduce a replacement gene.
PCT/GB1996/002357 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed WO1997011169A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
DE69637831T DE69637831D1 (en) 1995-09-21 1996-09-23 STRATEGY FOR IMPROVING THE EXPRESSION OF AN ENDOGENOUS GENE BY USING COMPOUNDS THAT CAN BIND TO THE NON-CODING REGIONS OF THE GENE TO BE BREATHED
US09/043,506 US6713457B2 (en) 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
EP96931887A EP0851918B1 (en) 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
CA2232738A CA2232738C (en) 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogenous gene
AU70896/96A AU7089696A (en) 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
NZ318818A NZ318818A (en) 1995-09-21 1996-09-23 The use of compounds that are able to bind to the non-coding regions of a gene in the manufacture of a medicament for suppressing the expression of an endogenous gene

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB9519299.3 1995-09-21
GBGB9519299.3A GB9519299D0 (en) 1995-09-21 1995-09-21 Genetic strategy

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US09/043,506 A-371-Of-International US6713457B2 (en) 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
US10/742,656 Division US20040254138A1 (en) 1995-09-21 2003-12-19 Suppression effectors that target non-coding regions and uses therefore

Publications (2)

Publication Number Publication Date
WO1997011169A2 WO1997011169A2 (en) 1997-03-27
WO1997011169A3 true WO1997011169A3 (en) 1997-06-12

Family

ID=10781063

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB1996/002357 WO1997011169A2 (en) 1995-09-21 1996-09-23 Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed

Country Status (10)

Country Link
US (2) US6713457B2 (en)
EP (1) EP0851918B1 (en)
AT (1) ATE422215T1 (en)
AU (1) AU7089696A (en)
CA (1) CA2232738C (en)
DE (1) DE69637831D1 (en)
ES (1) ES2321461T3 (en)
GB (1) GB9519299D0 (en)
NZ (1) NZ318818A (en)
WO (1) WO1997011169A2 (en)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9519299D0 (en) 1995-09-21 1995-11-22 Farrar Gwyneth J Genetic strategy
GB9604449D0 (en) * 1996-03-01 1996-05-01 Farrar Gwyneth J Genetic strategy ii
GB9606961D0 (en) * 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
US20040234999A1 (en) * 1996-04-02 2004-11-25 Farrar Gwenyth Jane Genetic suppression and replacement
US8551970B2 (en) * 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
EP0977841A2 (en) 1997-04-21 2000-02-09 University Of Florida Materials and methods for ribozyme treatment of retinal diseases
CA2287495A1 (en) * 1997-04-21 1998-10-29 University Of Florida Materials and methods for treatment of retinal diseases
US20040138168A1 (en) * 1999-04-21 2004-07-15 Wyeth Methods and compositions for inhibiting the function of polynucleotide sequences
IL145778A0 (en) * 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
WO2000066780A2 (en) 1999-04-30 2000-11-09 University Of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
GB0403600D0 (en) * 2004-02-18 2004-03-24 Trinity College Dublin Methods and reagents for treating disease
EP1764107A1 (en) * 2005-09-14 2007-03-21 Gunther Hartmann Compositions comprising immunostimulatory RNA oligonucleotides and methods for producing said RNA oligonucleotides
US8557787B2 (en) 2011-05-13 2013-10-15 The Board Of Trustees Of The Leland Stanford Junior University Diagnostic, prognostic and therapeutic uses of long non-coding RNAs for cancer and regenerative medicine

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993021202A1 (en) * 1992-04-15 1993-10-28 Yissum Research Development Company Of The Hebrew University Of Jerusalem Synthetic antisense oligodeoxynucleotides and pharmaceutical compositions containing them
WO1994003596A1 (en) * 1992-07-30 1994-02-17 University Of Hawaii Antisense viruses and antisense-ribozyme viruses
WO1994011494A1 (en) * 1992-11-09 1994-05-26 Thomas Jefferson University Antisense oligonucleotides to inhibit expression of mutated and wild type genes for collagen
WO1994022487A1 (en) * 1993-03-26 1994-10-13 Thomas Jefferson University Use of a col 1a1 mini-gene construct to inhibit collagen synthesis

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JPH0376580A (en) 1989-08-17 1991-04-02 Japan Tobacco Inc Escherichia coli manifestation vector and production of antiviral protein using the same
US5240846A (en) 1989-08-22 1993-08-31 The Regents Of The University Of Michigan Gene therapy vector for cystic fibrosis
US5223391A (en) * 1990-02-21 1993-06-29 President And Fellows Of Harvard College Inhibitors of herpes simplex virus replication
US5246921A (en) * 1990-06-26 1993-09-21 The Wistar Institute Of Anatomy And Biology Method for treating leukemias
NO312681B1 (en) 1990-08-24 2002-06-17 Univ California Process for the preparation of a pharmaceutical composition with suppressive action / activity
WO1992012262A1 (en) 1991-01-04 1992-07-23 Washington University Dna sequences related to isolated fragile x syndrome
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AU6786594A (en) 1993-05-11 1994-12-12 University Of North Carolina At Chapel Hill, The Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US6025127A (en) * 1994-01-14 2000-02-15 The Johns Hopkins University School Of Medicine Nucleic acid mutation detection in histologic tissue
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WO1994003596A1 (en) * 1992-07-30 1994-02-17 University Of Hawaii Antisense viruses and antisense-ribozyme viruses
WO1994011494A1 (en) * 1992-11-09 1994-05-26 Thomas Jefferson University Antisense oligonucleotides to inhibit expression of mutated and wild type genes for collagen
WO1994022487A1 (en) * 1993-03-26 1994-10-13 Thomas Jefferson University Use of a col 1a1 mini-gene construct to inhibit collagen synthesis

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Also Published As

Publication number Publication date
CA2232738C (en) 2010-11-23
ES2321461T3 (en) 2009-06-05
EP0851918A2 (en) 1998-07-08
NZ318818A (en) 2000-06-23
US20030096767A1 (en) 2003-05-22
EP0851918B1 (en) 2009-02-04
US20040254138A1 (en) 2004-12-16
CA2232738A1 (en) 1997-03-27
DE69637831D1 (en) 2009-03-19
US6713457B2 (en) 2004-03-30
ATE422215T1 (en) 2009-02-15
AU7089696A (en) 1997-04-09
WO1997011169A2 (en) 1997-03-27
GB9519299D0 (en) 1995-11-22

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